FDA News

FDA gives orphan drug designation for retinitis pigmentosa gene therapy

The FDA has given orphan drug designation to a gene therapy product intended for treatment of X-linked retinitis pigmentosa, Applied Genetic Technologies Corp. announced in a press release.

The drug candidate, which is intended specifically to treat X-linked retinitis pigmentosa caused by mutations in the RPGR gene, was granted orphan medicinal product designation for the same indication by the European Commission in June 2016, according to the release.

“We are on track to file an Investigational New Drug application for this program this year, and this orphan drug designation will help to accelerate the regulatory review process as we work to bring a new treatment option to XLRP patients,” Sue Washer, president and CEO of AGTC, said in the release.

 

The FDA has given orphan drug designation to a gene therapy product intended for treatment of X-linked retinitis pigmentosa, Applied Genetic Technologies Corp. announced in a press release.

The drug candidate, which is intended specifically to treat X-linked retinitis pigmentosa caused by mutations in the RPGR gene, was granted orphan medicinal product designation for the same indication by the European Commission in June 2016, according to the release.

“We are on track to file an Investigational New Drug application for this program this year, and this orphan drug designation will help to accelerate the regulatory review process as we work to bring a new treatment option to XLRP patients,” Sue Washer, president and CEO of AGTC, said in the release.